Drugs[JOURNAL]
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200 papers
Trastuzumab Rezetecan: First Approval.
Hoy SM
Drugs · 2026 Jan · PMID 41138046 · Publisher ↗
Trastuzumab rezetecan (Avida ) is a human epidermal growth factor receptor 2 (HER2)-directed antibody-drug conjugate being developed by Jiangsu HengRui Medicine Co., Ltd. for the treatment of various solid tumours, inclu...
Trastuzumab rezetecan (Avida ) is a human epidermal growth factor receptor 2 (HER2)-directed antibody-drug conjugate being developed by Jiangsu HengRui Medicine Co., Ltd. for the treatment of various solid tumours, including breast cancer, colorectal cancer, gastric cancer, gastroesophageal junction adenocarcinoma and lung cancer. In May 2025, it was approved by the National Medical Products Administration of China for the treatment of adults with unresectable locally advanced or metastatic non-small cell lung cancer (NSCLC) who have human epidermal growth factor receptor 2 (HER2) [ERBB2]-activating mutations and have received ≥ 1 previous systemic treatment. This article summarizes the milestones in the development of trastuzumab rezetecan leading to this first approval.
Correction: Senaparib: First Approval.
Lee A
Drugs · 2025 Nov · PMID 41091366 · Publisher ↗
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Sepiapterin: First Approval.
Lamb YN
Drugs · 2026 Jan · PMID 41091365 · Full text
An oral formulation of sepiapterin (Sephience™) is being developed by PTC Therapeutics for the treatment of phenylketonuria (PKU). Sepiapterin is a natural precursor of enzyme cofactor tetrahydrobiopterin (BH), which is...
An oral formulation of sepiapterin (Sephience™) is being developed by PTC Therapeutics for the treatment of phenylketonuria (PKU). Sepiapterin is a natural precursor of enzyme cofactor tetrahydrobiopterin (BH), which is required for phenylalanine hydroxylase (PAH)-mediated phenylalanine metabolism. Sepiapterin enhances PAH activity, which is deficient in patients with PKU. Sepiapterin received its first approval on 19 June 2025 in the European Union, for use in hyperphenylalaninaemia (HPA) in adult and paediatric patients with PKU. This was followed by approval in the USA on 28 July 2025, for the treatment of HPA in adult and paediatric patients ≥ 1 month of age with sepiapterin-responsive PKU. This article summarizes the milestones in the development of sepiapterin leading to its first approval for HPA in adult and paediatric patients with PKU.
Current and Emerging Treatments for Acid Sphingomyelinase Deficiency.
Silva Alves T, Fonseca Siqueira AL, Giugliani R
Drugs · 2025 Dec · PMID 41076598 · Publisher ↗
Acid sphingomyelinase deficiency is an ultra-rare disease characterised by generalised storage of sphingomyelin, caused by deficiency of the lysosomal enzyme acid sphingomyelinase, owing to the presence of biallelic path...
Acid sphingomyelinase deficiency is an ultra-rare disease characterised by generalised storage of sphingomyelin, caused by deficiency of the lysosomal enzyme acid sphingomyelinase, owing to the presence of biallelic pathogenic variants in the SMPD1 gene. The main disease manifestations are in the liver, spleen, lung and bone - with some patients having also involvement of the central nervous system. Patients show variable degrees of anaemia, thrombocytopenia and lipid abnormalities, among other findings. Clinically, acid sphingomyelinase deficiency spans from an acute neurovisceral form, with neurological involvement and early death (type A), to a chronic visceral disease, with no or minimal neurological manifestations (type B). An intermediate form, with chronic neurovisceral involvement, is presented by some patients (type A/B). Diagnosis involves the measurement of biomarkers, an assay of enzyme activity and genetic testing. Until a few years ago, treatment was mainly dependent on symptomatic management and bone marrow or solid organ (liver and/or lung) transplantation. In 2022, a specific enzyme replacement therapy with olipudase alfa was approved, and the results available indicate that it changed the therapeutic landscape for patients with acid sphingomyelinase deficiency type B and A/B. Research is being developed to address the needs of patients with acid sphingomyelinase deficiency type A, with gene therapy remaining as a promising approach.
Lisaftoclax: First Approval.
Syed YY
Drugs · 2025 Dec · PMID 41060518 · Publisher ↗
Lisaftoclax is a B-cell lymphoma 2 (BCL-2) inhibitor developed by Ascentage Pharma for the treatment of haematological malignancies. It is administered using a 5-day ramp-up schedule, reaching the target dose on day 6. L...
Lisaftoclax is a B-cell lymphoma 2 (BCL-2) inhibitor developed by Ascentage Pharma for the treatment of haematological malignancies. It is administered using a 5-day ramp-up schedule, reaching the target dose on day 6. Lisaftoclax received its first approval on 10 July 2025 in China for the treatment of chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who had received at least one prior systemic therapy, including a Bruton's tyrosine kinase (BTK) inhibitor. This article summarizes the milestones in the development of lisaftoclax leading to this first approval for CLL/SLL.
The Current Treatment Landscape for Congenital Adrenal Hyperplasia.
Tonge JJ, Bacila I, Krone NP
Drugs · 2025 Dec · PMID 41037194 · Publisher ↗
Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive endocrine disorders caused by impaired steroidogenesis and insufficient cortisol production from the adrenal cortex. The most common form of CAH is 2...
Congenital adrenal hyperplasia (CAH) is a group of autosomal recessive endocrine disorders caused by impaired steroidogenesis and insufficient cortisol production from the adrenal cortex. The most common form of CAH is 21-hydroxylase deficiency, caused by mutations in the CYP21A2 gene. This enzyme deficiency causes failure to progress through the steroidogenic pathways, consequently there is an increased synthesis of steroid hormone precursors that result in adrenal androgens excess, leading to virilisation in female patients. Over the past two decades, significant progress has been made in optimising corticosteroid replacement strategies, with a focus on reducing androgen excess while minimising glucocorticoid exposure. Modified-release hydrocortisone formulations, such as Efmody and Plenadren, as well as continuous subcutaneous infusion therapies, have been developed to help better mimic physiological cortisol rhythms. In addition, corticotropin-releasing hormone-1 (CRH1) receptor antagonists have been investigated and have now been approved as a novel approach to reducing adrenocorticotropic hormone (ACTH)-driven adrenal hyperplasia and androgen excess. More recently, promising novel approaches have been developed to control androgen excess and reduce glucocorticoid exposure. This review provides an overview of current standard-of-care treatments for CAH and highlights recent advancements in therapeutic strategies. By addressing the limitations of traditional glucocorticoid replacement, these innovations have the potential to improve long-term outcomes and quality of life for patients with CAH.
Advances in Pharmacotherapy for Menopausal Vasomotor Symptoms.
Young Moss S, Lee A, Simon JA
Drugs · 2025 Nov · PMID 41028653 · Full text
Vasomotor symptoms (VMS) are considered the cardinal symptoms of menopause, affecting up to 80% of American women at some point during the menopausal transition. VMS, particularly if they are moderate to severe, frequent...
Vasomotor symptoms (VMS) are considered the cardinal symptoms of menopause, affecting up to 80% of American women at some point during the menopausal transition. VMS, particularly if they are moderate to severe, frequent, or cause sleep disturbances, can have a negative impact on a woman's quality of life, physical and mental health, and professional life. Furthermore, VMS have been associated with negative health outcomes, including an increased risk of coronary heart disease and cognitive impairment. Menopausal hormonal therapy (MHT) is supported by level 1 evidence and can address many of the negative impacts associated with menopause, including VMS. However, not all women can take MHT (owing to having contraindications to their use) or choose not to take MHT. In addition to MHT, non-hormonal therapy options, which include neurokinin (NK)-targeted therapies, are also available. Fezolinetant (NK3 receptor antagonist) and the newly approved elinzanetant (NK1 and NK3 receptor antagonist) are non-hormonal treatment options approved for the treatment of VMS associated with menopause. These approvals expand the treatment options for women. A number of investigational agents are currently in phase 2 trials for potential future use for VMS; these include Q-122, PhytoSERM, NOE-115, GS1-144, and HS-10384. In this review, we highlight the recent advancements in our understanding of the pathophysiology of VMS and consider the current, new, and investigational treatment options for the treatment of VMS.
Mazdutide: First Approval.
Shirley M
Drugs · 2025 Dec · PMID 41028652 · Publisher ↗
Mazdutide (Xinermei) is a dual glucagon receptor (GcgR) and glucagon-like peptide-1 receptor (GLP-1R) agonist being developed by Eli Lilly and Company along with Innovent Biologics for use in weight management in adults...
Mazdutide (Xinermei) is a dual glucagon receptor (GcgR) and glucagon-like peptide-1 receptor (GLP-1R) agonist being developed by Eli Lilly and Company along with Innovent Biologics for use in weight management in adults with obesity or overweight and for the treatment of type 2 diabetes (T2D). In June 2025, mazdutide received its first approval, in China, for use (in combination with diet control and increased physical activity) in long-term body weight management in adults with a body-mass index (BMI) of ≥ 28 kg/m or with a BMI ≥ 24 kg/m together with one or more weight-related comorbidity. Subsequently, in September 2025, mazdutide also received approval in China for use in glycaemic control in adults with T2D. Additionally, mazdutide is under clinical evaluation for use in the treatment of metabolic dysfunction-associated fatty liver disease, obstructive sleep apnoea and alcohol use disorder. This article summarises the milestones in the development of mazdutide leading to this first approval for long-term body weight management in adults with obesity or overweight.
Ifupinostat: First Approval.
Fung S
Drugs · 2025 Dec · PMID 41028651 · Full text
Ifupinostat (; Betlin) is a first-in-class dual phosphoinositide 3-kinase α (PI3Kα)/histone deacetylase (HDAC) inhibitor being developed by BeBetter Med for the treatment of relapsed or refractory diffuse large B-cell ly...
Ifupinostat (; Betlin) is a first-in-class dual phosphoinositide 3-kinase α (PI3Kα)/histone deacetylase (HDAC) inhibitor being developed by BeBetter Med for the treatment of relapsed or refractory diffuse large B-cell lymphoma. On the 30th June 2025, ifupinostat received its first approval in China as a monotherapy for adults with relapsed or refractory diffuse large B-cell lymphoma who have received at least two lines of systemic therapy. Continued approval is dependent on the results of a confirmatory, randomized, controlled phase 3 trial. This article summarizes the milestones in the development of ifupinostat leading to this first approval as a monotherapy for adults with relapsed or refractory diffuse large B-cell lymphoma who have received at least two lines of systemic therapy.
Long-Term Toxicities of Immune Checkpoint Inhibitors.
Abraham PE, Johnson DB
Drugs · 2025 Dec · PMID 41028650 · Full text
Immune checkpoint inhibitors (ICIs) produce often durable responses in many cancer types but are associated with autoimmune-like toxicities. These immune-related adverse events (irAEs) occur in multiple organ systems and...
Immune checkpoint inhibitors (ICIs) produce often durable responses in many cancer types but are associated with autoimmune-like toxicities. These immune-related adverse events (irAEs) occur in multiple organ systems and often improve with steroids or therapy cessation. However, many irAEs have either partial or no improvement, leading to residual sequelae even after ICI discontinuation. Chronic irAEs, often defined as irAEs lasting > 3 months, may occur in up to 40-50% of patients treated with ICI therapy and have a wide range of manifestations, severity, and duration. These chronic events likely stem from either ongoing inflammation or sequalae of prior inflammatory-mediated damage. IrAEs impacting the endocrine glands and joints have particularly high rates of chronicity, while chronic events impacting the gastrointestinal (GI) tract, liver, and lungs are less common. In this review, we discuss updated studies surrounding chronic irAEs, providing an overview, followed by organ-specific considerations and consideration of future directions. Key challenges for the field include characterizing the chronic impact of irAEs, developing better treatment or prevention strategies (one possible solution being anti-cytokine therapy), and identifying which patients have active inflammation.
Clinical Efficacy, Safety and Imaging Effects of Oral Valiltramiprosate in APOEε4/ε4 Homozygotes with Early Alzheimer's Disease: Results of the Phase III, Randomized, Double-Blind, Placebo-Controlled, 78-Week APOLLOE4 Trial.
Abushakra S, Power A, Watson D … +17 more , Porsteinsson A, Sabbagh M, MacSweeney E, Cohen S, Boada Rovira M, Doraiswamy PM, Liang E, Flint S, Kesslak JP, McLaine R, Albayrak A, Schaefer J, Yu J, Tolar L, Dickson S, Hey JA, Tolar M
Drugs · 2025 Nov · PMID 41015981 · Full text
BACKGROUND: The apolipoprotein E ε4 (APOE ε4) allele is the strongest genetic risk factor for Alzheimer's disease (AD), with homozygotes accumulating a high burden of cerebral beta-amyloid (Aβ) pathology. Valiltramiprosa...
BACKGROUND: The apolipoprotein E ε4 (APOE ε4) allele is the strongest genetic risk factor for Alzheimer's disease (AD), with homozygotes accumulating a high burden of cerebral beta-amyloid (Aβ) pathology. Valiltramiprosate/ALZ-801 is a small-molecule potent inhibitor of Aβ-oligomer formation. The efficacy, safety/tolerability, and brain volume effects of oral valiltramiprosate were evaluated in this phase III, randomized, double-blind, placebo-controlled, multi-center, 78-week trial in homozygotes with early symptomatic AD. METHODS: The study enrolled eligible APOE4/4 subjects aged 50-80 years with Early AD (Mini-Mental State Examination [MMSE] 22-30), which included mild cognitive impairment (MCI) and mild dementia, Clinical Dementia Rating-Global Score (CDR-G) of 0.5 or 1, who were randomized 1:1 to valiltramiprosate (265 mg twice/day) or placebo. The primary outcome was AD Assessment Scale-Cognitive Subscale (ADAS-Cog13); the key secondary outcomes were CDR-Sum of Boxes (CDR-SB) and Amsterdam-Instrumental Activities of Daily Living (IADL), and a secondary outcome was Disability Assessment for Dementia (DAD). The main imaging outcome was hippocampal volume on MRI; diffusion tensor imaging (MRI-DTI) assessed microstructural tissue integrity. Amyloid-related imaging abnormalities (ARIA) were monitored with MRIs every 26 weeks. RESULTS: A total of 325 participants enrolled and received study drug. At 78 weeks, the overall efficacy population did not show significant effects on ADAS-Cog13 or other clinical outcomes compared with placebo (ADAS-Cog13: 11% slowing; p = 0.607, N = 320), but showed significant slowing of hippocampal atrophy (18%, p = 0.017, N = 290). Prespecified analyses by disease severity (stratification variable) showed no significant clinical effects in mild AD (MMSE ≤26, N = 195). The prespecified MCI group (MMSE >26, N = 125) showed nominally significant positive effects on ADAS-Cog13 (52%, nominal p = 0.041) and DAD (96%, nominal p = 0.016), positive trend on CDR-SB (102%, nominal p = 0.053), with significant hippocampal atrophy slowing (26%, p = 0.004), and positive grey/white matter effects on MRI-DTI. In the MCI group, positive ADAS-Cog13 drug effects showed significant subject-level correlations with positive effects on imaging outcomes. The most common adverse events were nausea, vomiting, and decreased appetite (more than double placebo rate), with no increased risk of brain edema or microhemorrhages. CONCLUSIONS: The APOE4/4 Early AD population did not show significant clinical efficacy at 78 weeks but showed significant brain atrophy slowing. Prespecified analyses at the MCI stage showed nominally significant slowing of clinical decline with significant hippocampal atrophy slowing. Oral valiltramiprosate may provide a favorable benefit-risk profile and simple treatment paradigm for homozygotes with MCI. These results will inform the design of future MCI trials. TRIAL REGISTRATION: Clinicaltrials.gov: NCT04770220; EudraCT Number: 2020-005755-20.
Sebetralstat: First Approval.
Blair HA
Drugs · 2025 Nov · PMID 41014446 · Publisher ↗
Sebetralstat (EKTERLY), an orally available plasma kallikrein inhibitor, is being developed by KalVista Pharmaceuticals for the on-demand treatment of acute attacks of hereditary angioedema (HAE). On 7 July 2025, sebetra...
Sebetralstat (EKTERLY), an orally available plasma kallikrein inhibitor, is being developed by KalVista Pharmaceuticals for the on-demand treatment of acute attacks of hereditary angioedema (HAE). On 7 July 2025, sebetralstat received its first approval in the USA for the treatment of acute attacks of HAE in adult and pediatric patients aged 12 years and older. The drug has since been approved on 15 July 2025 in the UK for the treatment of HAE attacks in adults and adolescents aged 12 years and older. Sebetralstat has also received a positive opinion in the EU for the symptomatic treatment of acute attacks of HAE in adults and adolescents aged 12 years and older. This article summarizes the milestones in the development of sebetralstat leading to this first approval for HAE.
Onradivir: First Approval.
Lee A
Drugs · 2025 Dec · PMID 40996478 · Publisher ↗
Onradivir (, Anruiwei) is a small molecule RNA polymerase inhibitor that potently binds to the PB2 cap-binding domain of RNA polymerase to inhibit the replication of the influenza A virus. It was developed by Guangdong R...
Onradivir (, Anruiwei) is a small molecule RNA polymerase inhibitor that potently binds to the PB2 cap-binding domain of RNA polymerase to inhibit the replication of the influenza A virus. It was developed by Guangdong Raynovent Biotech and received its first approval in May 2025 in China based on results from the NCT04683406 phase III trial. This article summarizes the milestones in the development of onradivir leading to this first approval for the treatment of uncomplicated influenza A in adults, excluding those at high risk for influenza-related complications.
Famitinib: First Approval.
Keam SJ
Drugs · 2025 Dec · PMID 40996477 · Publisher ↗
Famitinib (), an oral, multi-targeting tyrosine kinase inhibitor (TKI) with dual anti-tumour effects (inhibition of both cell proliferation and angiogenesis), is being developed by Jiangsu Hengrui Medicine Co., Ltd for t...
Famitinib (), an oral, multi-targeting tyrosine kinase inhibitor (TKI) with dual anti-tumour effects (inhibition of both cell proliferation and angiogenesis), is being developed by Jiangsu Hengrui Medicine Co., Ltd for the treatment of solid tumours. In May 2025, famitinib was granted conditional approval for use in combination with camrelizumab for the treatment of patients with recurrent or metastatic cervical cancer who have failed prior platinum-based chemotherapy and have not received prior bevacizumab in China. This article summarizes the milestones in the development of famitinib leading to this first approval for the treatment of recurrent or metastatic cervical cancer.
JAK Inhibitors for the Treatment of Vitiligo: Current Evidence and Emerging Therapeutic Potential.
Ferreira C, King B, Torres T
Drugs · 2025 Dec · PMID 40996476 · Publisher ↗
Vitiligo is a common, chronic, immune-mediated disorder characterized by progressive skin depigmentation, often associated with significant psychosocial burden and impaired quality of life. Therapeutic management remains...
Vitiligo is a common, chronic, immune-mediated disorder characterized by progressive skin depigmentation, often associated with significant psychosocial burden and impaired quality of life. Therapeutic management remains challenging, with limited effective options available. Although topical corticosteroids, calcineurin inhibitors, and narrowband ultraviolet B (NB-UVB) phototherapy constitute the mainstays of treatment, many patients, particularly those with extensive or refractory disease, fail to achieve satisfactory or durable repigmentation. The clinical course is further complicated by high relapse rates and heterogeneous treatment responses across different anatomical sites. Recent advances in the understanding of vitiligo pathogenesis have identified the Janus kinase/signal transducer and activator of transcription (JAK/STAT) pathway as a central driver of immune-mediated melanocyte destruction. This pathway is activated by key cytokines involved in vitiligo, including interferon gamma (IFN-γ), interleukin 15 (IL-15), among others, which sustain cytotoxic T cell infiltration and melanocyte apoptosis. As a result, JAK inhibitors have emerged as promising targeted therapies for vitiligo. Several topical and JAK inhibitors are currently under clinical investigation, with one topical agent, ruxolitinib cream, already approved for the treatment of vitiligo. Topical ruxolitinib, a JAK1/2 inhibitor, has demonstrated consistent and clinically meaningful repigmentation, particularly in facial lesions, and is already approved for use in both adolescents and adults. Among oral agents, ritlecitinib (a JAK3/tyrosine kinase expressed in hepatocellular carcinoma (TEC) inhibitor), upadacitinib and povorcitinib (JAK1 inhibitors) have shown the most promising efficacy, either as monotherapy or in combination with NB-UVB phototherapy. Ongoing phase III trials are expected to further define their role in clinical practice. Other agents, including tofacitinib, baricitinib, abrocitinib, among others, are currently under investigation or being used off-label in clinical practice. JAK inhibitors exhibit variable safety profiles depending on selectivity, formulation, and dose. Topical agents are generally well tolerated with minimal systemic absorption, whereas oral JAK inhibitors require monitoring owing to potential risks of infection, hematologic abnormalities, and cardiovascular events. In this article, we review the current evidence on the efficacy and safety of topical and oral JAK inhibitors for vitiligo and contextualize their role within the broader landscape of emerging therapeutic strategies.
Fosrolapitant/Palonosetron: First Approval.
Fung S
Drugs · 2025 Nov · PMID 40991189 · Publisher ↗
Fosrolapitant/palonosetron (Ritanine; ) is a fixed-dose combination of fosrolapitant, a novel neurokinin-1 (NK-1) receptor antagonist prodrug, and palonosetron, a second generation 5-hydroxytryptamine-3 (5-HT3) receptor...
Fosrolapitant/palonosetron (Ritanine; ) is a fixed-dose combination of fosrolapitant, a novel neurokinin-1 (NK-1) receptor antagonist prodrug, and palonosetron, a second generation 5-hydroxytryptamine-3 (5-HT3) receptor antagonist. It is being developed by Fujian Shengdi Pharmaceutical as a preventative treatment of chemotherapy-induced nausea and vomiting. On the 29 of May 2025, fosrolapitant/palonosetron received its first approval in China for the prevention of acute and delayed nausea and vomiting caused by highly emetogenic chemotherapy in adults. This article summarizes the milestones in the development of fosrolapitant/palonosetron leading to this first approval in chemotherapy-induced nausea and vomiting.
Pharmacological Therapies in Paroxysmal Nocturnal Haemoglobinuria: Focus on Complement Inhibition.
Kelly RJ, Holt M, Szer J
Drugs · 2025 Nov · PMID 40986193 · Full text
Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare acquired genetic stem cell disorder based on a mutation in the PIGA gene that results in susceptibility of resulting blood cells to complement-mediated intravas...
Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-rare acquired genetic stem cell disorder based on a mutation in the PIGA gene that results in susceptibility of resulting blood cells to complement-mediated intravascular haemolysis (IVH). In countries where anti-complement therapy is available, pharmacological treatments have transformed this disease from a highly morbid and sometimes lethal disorder. The first treatment developed was the terminal complement (C5) monoclonal antibody inhibitor eculizumab, in 2002. This has been largely supplanted by a longer-acting antibody, ravulizumab, targeting the same binding site on C5. These agents significantly modify the natural history of the disease by reducing the risk of thrombosis, the most lethal complication of PNH, as well as reducing transfusion dependence and improving renal function, quality of life and probably, survival. Other terminal inhibitors available include eculizumab biosimilars, crovalimab, pozelimab and cemdisiran (combination). Despite this, a proportion of patients develop extravascular haemolysis (EVH) based on the accumulation of C3 components on these PNH blood cells, which no longer undergo IVH because of C5 inhibition. This has led to the development of proximal complement inhibitors, which have been generally successful at reducing this iatrogenic complication, improving haemoglobin concentrations, reducing transfusion dependency and improving quality of life. Currently available proximal inhibitors (and their targets) are pegcetacoplan (C3), danicopan (Factor D) and iptacopan (Factor B). While effective, as with all other complement inhibitors, there is a risk of breakthrough IVH with their use and approaches to manage this complication are being developed.
The Road to Remyelination in Multiple Sclerosis: Breakthroughs, Challenges, and Considerations for Future Trial Design.
Zuroff L, Farkhondeh V, Bove R … +1 more , Green AJ
Drugs · 2025 Nov · PMID 40956548 · Full text
Despite major advances in multiple sclerosis (MS) treatment, disability accumulation independent of relapse activity remains a significant challenge. Chronic demyelination is a key driver of neurodegeneration and disease...
Despite major advances in multiple sclerosis (MS) treatment, disability accumulation independent of relapse activity remains a significant challenge. Chronic demyelination is a key driver of neurodegeneration and disease progression, highlighting remyelination as a promising therapeutic strategy. Collective evidence from several phase II clinical trials now indicates that remyelination is feasible in patients with MS. However, several drug development programs have yielded less robust responses than anticipated, which has limited translation of therapies into clinical practice. This underscores the need for refined trial methodologies, including careful selection of patient populations, validation of biomarkers, and implementation of functional outcomes that accurately capture remyelination effects. In this review, we summarize the current understanding of remyelination mechanisms, assess the therapeutic landscape, and discuss strategies to improve clinical trial design. Addressing key questions-such as the optimal timing, patient selection, and methods of measurement-will be crucial for advancing the field and ushering in a new wave of MS therapeutics.
Update on Medical Treatment of Cushing's Syndrome.
Dillon BR, Agrawal N, Schwarz Y … +5 more , Dancel-Manning K, Tabarin A, Lacroix A, Hofland LJ, Feelders RA
Drugs · 2025 Oct · PMID 40954428 · Full text
First-line treatment of endogenous Cushing's syndrome (CS) is surgical removal of the tumor responsible for cortisol excess. However, medical therapy has an established role in treatment when patients are not surgical ca...
First-line treatment of endogenous Cushing's syndrome (CS) is surgical removal of the tumor responsible for cortisol excess. However, medical therapy has an established role in treatment when patients are not surgical candidates or decline surgery, residual or recurrent disease is present and not amenable to repeat resection, and control of hypercortisolism is needed either preoperatively or while awaiting the effects of radiotherapy. The approach to medical therapy should be tailored based on the etiology, degree of hypercortisolism, and patient characteristics. Currently available medical therapy for all etiologies of CS either blocks adrenal production of cortisol or blocks its action at the level of the glucocorticoid receptor. Currently available medical therapy for Cushing's disease (CD) targets the adrenocorticotropic hormone-secreting pituitary tumor through activation of somatostatin and dopamine receptors, alkylating DNA damage, or immune system activation. More focused therapy with greater efficacy and fewer adverse effects is needed, particularly in the case of CD, with potential targets and drugs identified and in development.
The End of the Line for Modified-Release Opioids in Acute Pain Management: How Did We Get Here?
Macintyre PE, Stevens JA, Quinlan J
Drugs · 2025 Dec · PMID 40932636 · Publisher ↗
Following announcements from professional and governmental bodies across a number of countries, modified-release (MR) opioids are no longer recommended in the routine management of acute pain, and so should not be initia...
Following announcements from professional and governmental bodies across a number of countries, modified-release (MR) opioids are no longer recommended in the routine management of acute pain, and so should not be initiated for this reason. The recommendations are not new, but a recent cluster of publications by key professional and governmental bodies has more clearly challenged their use and highlighted the need for change in guideline-driven and individual practice. The inclusion of MR opioids in many postoperative pain regimens relatively soon after they were first marketed for use in patients with chronic non-cancer pain, was not based on sound evidence, and there remains no evidence of benefit. In contrast, however, good evidence has accumulated that shows they not only provide less effective pain relief compared with immediate-release opioids, but that they lead to a higher risk of adverse effects including opioid-induced ventilatory impairment and persistent opioid use.
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